Orphan Drug Designation for HT-100 for Duchenne Muscular Dystrophy

Halo Therapeutics announced that the FDA has granted orphan drug designation for HT-100 (halofuginone) for Duchenne muscular dystrophy (DMD). Plans to initiate a Phase 2 trial in 2012 are underway. DMD is a progressive and fatal neuromuscular disorder caused by a mutation in the dystrophin gene, resulting in progressive muscle weakness. The disease manifests itself first in weakened skeletal muscles and eventually results in cardiac and pulmonary impairment.

HT-100 is an orally available small-molecule drug candidate that exerts its therapeutic effects by inhibiting the pathological fibrotic process in muscle and directly stimulating healthy muscle-fiber regeneration.